Testing a new approach to prevent graft-versus-host disease after stem cell transplantation

Randomized Phase 2 Study Testing Two Conditioning Regimen With a Single Prophylaxis of Graft-versus-host Disease by Cyclophosphamide and Methotrexate Post-transplant in Patients Eligible for Matched-donor Allograft Transplantation

Phase 2 Interventional Nantes University Hospital · NCT06252870

This study is testing a new treatment to see if a combination of two drugs can help prevent graft-versus-host disease in patients receiving stem cell transplants, allowing them to stop taking immunosuppressive drugs sooner.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment82 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorNantes University Hospital Academic / other
Drugs / interventionschemotherapy, cyclophosphamide, methotrexate
Locations3 sites (Angers and 2 other locations)
Trial IDNCT06252870 on ClinicalTrials.gov

What this trial studies

This study aims to evaluate the effectiveness of a combination of high-dose post-transplant cyclophosphamide and methotrexate in preventing graft-versus-host disease (GVHD) in patients undergoing allogeneic hematopoietic stem cell transplantation with reduced-intensity conditioning. The approach seeks to allow for the early discontinuation of immunosuppressive drugs, which typically are required for several months post-transplant. Participants will receive these treatments on specific days following their transplant, and the study will compare outcomes to standard care. The goal is to improve patient outcomes and reduce the burden of long-term immunosuppression.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 to 70 with hematologic malignancies who are eligible for reduced-intensity conditioning and have a compatible HLA donor.

Not a fit: Patients with a history of allograft, those eligible for myeloablative conditioning, or with other progressive cancers may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly reduce the incidence of GVHD and the need for prolonged immunosuppressive therapy in transplant patients.

How similar studies have performed: Previous studies have shown promising results with similar approaches in preventing GVHD, particularly in the context of haploidentical transplants, but this specific combination in reduced-intensity conditioning is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Age: ≥ 18 and ≤ 70 years old
* Patient with hematologic malignancy
* Indication for HSC allograft with attenuated conditioning
* Pluripotent stem cell (PSC) engraftment
* Availability of a 10/10 familial or non-familial HLA compatible donor
* Consent to the protocol
* ECOG \<=2
* Woman of childbearing age with negative pregnancy test and on highly effective contraception during treatment and for a period of 12 months after stopping MTX and CY
* Man of childbearing age with highly effective contraception during treatment and for a period of 6 months after stopping MTX and CY and a period of 12 months after stopping MTX and CY if TBF conditioning regimen arm
* Negative Hepatitis B, C, HIV serologies
* Social security affiliation

Exclusion Criteria:

* History of allograft
* Patient eligible for myeloablative conditioning (MAC)
* Bone marrow transplant
* Other progressive cancerous disease, or antecedent of cancer in the last five years, with the exception of a carcinoma of the skin or a carcinoma in situ of the uterine cole treated and in remission.
* Progressive psychiatric condition
* Pregnant or breastfeeding woman,
* Woman or man of childbearing age with lack of effective contraception
* Serious and uncontrolled concomitant infection
* Cardiac: systolic ejection fraction \< 50% by transthoracic ultrasound or by isotopic method (isotope gamma angiography), NYHA II, III or IV heart failure, active rhythmic, valvular or ischemic heart disease or anteriority
* Respiratory with EFR: DLCOc \<40% of theoretical
* Renal: creatinine clearance \< 50 ml/min (assessment with MDRD method)
* Urological: active urinary tract infection, history of acute urothelial toxicity due to cytotoxic chemotherapy or radiotherapy, known obstruction of urinary flow, pre-existing hemorrhagic cystitis
* Hepatic: transaminases greater than 5 times normal or bilirubin greater than 2 times normal
* Person protected by law (major under guardianship, curatorship or legal protection)
* Vaccination against yellow fever in the last year
* Known or suspected hypersensitivity to rabbit proteins as well as to the active substance and excipients of all investigational and ancillary drugs administered during the study,
* Contraindication to any of the investigational or adjuvant drugs administered during the study
* Patient not speaking French

Where this trial is running

Angers and 2 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Graft Versus Host DiseaseHematologic MalignancyHematopoietic stem cell allograftMethotrexatePost-transplant cyclophosphamide
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.